Sarepta Therapeutics, Inc. Q3 2020 Earnings Conference Call
Forward Looking Statements
Thursday, November 5, 2020 - 4:30 p.m. Eastern Time
In order to provide Sarepta's investors with an understanding of its current results and future prospects, forward-looking statements will be made during this conference call. Any statements made by Sarepta that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "anticipate," "plan," "expect," "will," "may," "intend," "prepare," "look," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our future operations, financial performance and projections, business plans, market opportunities, priorities and research and development programs including: SRP-9001's goal to treat Duchenne muscular dystrophy by safely delivering to skeletal, diaphragm and cardiac muscle a gene that robustly codes for a truncated but functional form of the structural protein dystrophin called microdystrophin; Study 102's goal to show in a well-controlled trial that SRP-9001 is safe and effective in children with DMD; the potential of our commercial process and manufacturing capacity for SRP-9001 to provide us capability to produce and launch a commercial product to fully serve the community; the goal and design of study 103; LGMD2E's potential read through to our remaining LGMD portfolio and supportive confirmatory read through to our SRP- 9001 program for DMD; the expectation that the brand name for casimersen will be AMONDYS 45; the expected PDUFA date of February 25, 2021; the expectation to have three approved therapies capable, together, of treating nearly 30% of the DMD community, if we obtain approval for casimersen; the potential of our product candidates, including our RNA technology's potential to bring therapies to as many as about 85% of patients living with DMD, and PPMO's potential to profoundly improve the efficacy and convenience of our RNA technology and to substantially increase dystrophin production; expected plans and milestones, including commencing enrollment and dosing a trial with commercial process material (study 103) in 2020, having the last one-year visit for study 102 in December 2020 and releasing results from study 102 in the first quarter of 2021, our plan to speak with the FDA and start study 301 in 2021 after we have data available from study 103, our plan to design and propose to the Division for their review and input additional studies including in older and non-ambulant patients, our plan to engage with the FDA on the design of a pivotal study for LGMD2E, providing an update on safety, systemic exposure and exon skipping for our PPMO SRP-5051 candidate at 20 mgs/kg by the end of 2020, our intention with respect to SRP-5051, if safety signals permit, to continue to dose up to 40 mgs/kg early next year, and ultimately potentially even higher, providing an update on our entire pipeline, both gene therapy and RNA, in 2021, and our plan to initiate a proof-of-concept in vivo study in collaboration with USAMRIID for COVID-19.
These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; our dependence on certain manufacturers to produce our products and product candidates, including any inability on our part to accurately anticipate product demand and timely secure manufacturing capacity to meet product demand, may impair the availability of product to successfully support various programs; our data for casimersen, SRP-5051,SRP-9001, the LGMD programs and/or other programs may not be sufficient for obtaining regulatory approval; success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; the expected benefits and opportunities related to our agreements with our strategic partners may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreements, challenges and uncertainties inherent in product research and development and manufacturing limitations; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, many of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2019 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. You should not place undue reliance on forward-looking statements. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except to the extent required by applicable law or SEC rules.